Should Primary Progresssive MS be an Orphan Disease

We are discussing how we get treatments to progressive MSers. We need the Big Pharma Alternative and a change in view

Currently there are no approved DMT for progressive MS and when trials are done, most times the trials are done in Secondary Progressive MS. This is because there are more people with SPMS than PPMS and so trials are easier to recruit and do.

However, as we all know PPMSers feel alienated and let down by the medical profession. Could we make an incentive to do studies in PPMSers.

In the United States, the Rare Diseases Act of 2002 defines rare disease strictly according to prevalence, specifically “any disease or condition that affects less than 200,000 persons in the United States, or about 1 in 1,500 people. This definition is essentially like that of the Orphan Drug Act of 1983, a federal law that was written to encourage research into rare diseases and possible cures. In Japan, the legal definition of a rare disease is one that affects fewer than 50,000 patients in Japan, or about 1 in 2,500 people. The European Commission on Public Health defines rare diseases as “life-threatening or chronically debilitating diseases which are of such low prevalence that special combined efforts are needed to address them. The term low prevalence is later defined as generally meaning fewer than 1 in 2,000 people.

MS is no longer going to get orphan status in the US with 250,000 affected and a multi-billion dollar market. If a case could be made for progressive MS there would be less than 200,000 people affected and there is no available treatment. 

Can you lobby for this status because it lowers the bar for getting drugs to you. Only one major trial is needed so those academic led studies become more feasible, one trial cuts cost so pharma will be more willing to take a punt. 

Should the MS Community have another go at this?

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  • I could see how that might shortcut the process to finding more effective neuroprotective drugs for the people who need them most. Once something effective was found, it would quickly (I hope) be prescribed off label to those with SPMS and even RRMSers. Sounds like an excellent approach. Or am I missing something?

    • You need to get a focus group possibly within the MS Societies to lobby the regulators or to lobby politicians to lobby the regulators…

    • You are missing the point.

      The reason why there re no drugs for progressive MS was the realisation that it may be a different process than RRMS, as you suggest, but most importantly the trial design is not developed enough to easily detect an effect and then importantly time and money.

      You can lump progressive MS together to be an orphan disease.

      The point is that as an orphan disease it lowers the bar for people/company to invest in these areas so maybe they will chance doing a study in progressive MS first rather than doing RRMS trials and then much later doing progressive MS.

      In terms of epiphany we don't need one, we have more ideas than we can handle, if you have to do two 1000 person trials taking 6-7 years who has the resource to do it?….Pharma

      If they cannot make money they are not going to invest millions in developing an old drug…it is simple phamacoeconomics.

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